A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

NCT06789445 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: OpCT-001-101
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 4

Brief Summary

Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Conditions

Primary Photoreceptor Disease; Retinitis Pigmentosa (RP); Usher Syndrome; Inherited Retinal Disease (IRD); Rod-Cone Dystrophy; Rod-Cone Disease; Retinal Degeneration; Cone-Rod Disease (C-RD); Cone-Rod Dystrophy

Keywords

CLARICO; Photoreceptor cells; Cell Therapy; Cellular Therapy; Usher Syndrome; Retinitis Pigmentosa; Inherited retinal disease (IRD); Primary Photoreceptor disease (PPD); Rod-Con Disease (R-CD); Cone-Rose disease (C-RD)

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit

  From OpCT-001 administration through the Week 52 visit

Secondary Outcomes (1)

• Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT)

  From baseline through the Week 52 visit

Study Arms (4)

Cohort 1

EXPERIMENTAL

OpCT-001 dose level 1 will be administered via subretinal injection.

Biological: OpCT-001

Cohort 2

EXPERIMENTAL

OpCT-001 dose level 2 will be administered via subretinal injection.

Biological: OpCT-001

Cohort 3 (optional)

EXPERIMENTAL

OpCT-001 dose level 3 will be administered via subretinal injection.

Biological: OpCT-001

Cohort 4

EXPERIMENTAL

OpCT-001 dose level 4 will be administered via subretinal injection.

Biological: OpCT-001

Interventions

OpCT-001 BIOLOGICAL

Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).

Cohort 1

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed genetic diagnosis of primary photoreceptor (PR) disease
• Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.
• Retinal structure examination in the study eye demonstrating regions suitable for cell administration.

You may not qualify if:

• Clinically relevant, active ocular inflammation or infection
• Glaucoma or other significant optic neuropathy
• Diabetic macular edema or diabetic retinopathy
• Clinically significant cystoid macular edema
• In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia
• Ocular surgery ≤3 months before Screening
• Monocular vision (ie, no light perception in the fellow eye)
• Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.
• Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression
• History of any cell therapy, gene therapy, or retinal implant at any time
• Previously received a bone marrow or solid organ transplant

Sponsors & Collaborators

• BlueRock Therapeutics: lead

Study Sites (4)

University of Miami, Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

RECRUITING

Mid Atlantic Retina/ Wills Eye

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

RECRUITING

University of Wisconsin

Madison, Wisconsin, 53705, United States

RECRUITING

Related Links

• Related Info

MeSH Terms

Conditions

Retinitis Pigmentosa; Usher Syndromes; Cone-Rod Dystrophies; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Deaf-Blind Disorders; Deafness; Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Hearing Loss, Sensorineural; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Blindness; Vision Disorders; Abnormalities, Multiple; Congenital Abnormalities; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Patient Engagement

CONTACT

1-877-380-3931; clinicaltrials@bluerocktx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Phase 1: None (Open Label) Phase 2: Investigators and study site personnel outside of the surgical team will be masked to dose level assignments
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 1 of the study will include up to 4 planned dose levels to be administered across up to 4 cohorts. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on the Phase 1 safety and tolerability data.

Study Record Dates

• First Submitted: January 17, 2025
• First Posted: January 23, 2025
• Study Start: March 10, 2025
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2030
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

US (4)